Results 71 to 80 of about 222,490 (305)

Early and accelerated access programs for medical devices in the European Union: mapping regulatory derogations and national schemes

Frontiers in Medical Technology
BackgroundEarly access programs (EAPs) can provide patients with medical devices before full market access in urgent or high unmet-need situations. Unlike pharmaceuticals, the European Union (EU) has no harmonised early-access framework for medical ...
Baptiste Haon, Baptiste Haon, Mira Hartmann, Mira Hartmann, Sanae Akodad, Hilde Stevens, Lise Rochaix   +6 more
doaj   +1 more source

Safety and Effectiveness of Sutimlimab in Cold Agglutinin Disease: A Real‐World International Experience

American Journal of Hematology, EarlyView.
ABSTRACT Sutimlimab is a monoclonal antibody against complement fraction C1s approved for the treatment of hemolytic anemia due to cold agglutinin disease (CAD). Here, we analyzed and report the largest international CAD cohort of sutimlimab‐treated patients ever reported to highlight its safety and effectiveness in the real‐world setting. We accrued a
Bruno Fattizzo, Elisa Sambruna, Yoshitaka Miyakawa, Marc Michel, Carlo Visco, Michael Fillitz, George Kannourakis, Maria Bruna Greve, Moritz Kleemiss, Johan Cid, Andrea Patriarca, Laura Pavan, Arbana Dizdari, Nilla Maschio, Antonella Sau, Cinzia Bitetti, Riccardo Mario Colombo, Luigi Curreli, Dorela Lame, Uros Markovic, Fabrizio Pappagallo, Giacinto Luca Pedone, Davide Rapezzi, Vincenzo Sammartano, Nicola Tumedei, Antonio Giovanni Solimando, Jalink Marit, Luana Fianchi, Jahanzaib Khwaja, Antonio De Vivo, Francesco Lanza, Fabrizio Vianello, Esther N. Oliva, Catherine Broome, Francesco Passamonti, Ulrich Jaeger, Bernd Jilma, Guillaume Moulis, Wilma Barcellini   +38 more
wiley   +1 more source

Safety considerations in the evolving legal landscape of psychedelic-assisted psychotherapy

Substance Abuse Treatment, Prevention, and Policy, 2022
International drug policy is rapidly evolving in tandem with promising evidence for psychedelic-assisted psychotherapy (PAP) in treating a range of mental health conditions.
Victor Mocanu, Lindsay Mackay, Devon Christie, Elena Argento   +3 more
doaj   +1 more source

The 9th International RASopathies Symposium

American Journal of Medical Genetics Part A, EarlyView.
ABSTRACT The RASopathies are a group of congenital disorders with overlapping clinical manifestations that are caused by pathogenic germline or early somatic variants that result in the hyperactivation of the RAS/mitogen‐activated protein kinase (MAPK) signaling pathway.
Pau Castel, Lisa Schoyer, Beth Stronach, Raya Bogdanova, Anton M. Bennett, Jaishri Blakeley, Miriam Bornhorst, Tammy Bowers, Saskia M. Brachmann, Emma Burkitt‐Wright, Kathryn Chatfield, Alessandro De Luca, Khalil El‐Chammas, Abdul Elkadri, John E. Fortunato, Bruce D. Gelb, Anne Goriely, Karen Gripp, Kassidy Grover, Lindsay Homan, Kenneth A. Kern, Maija Kiuru, Charles (Chuck) Lawson, Yong‐Seok Lee, Frank McCormick, Gina Ney, Cristina Nuevo‐Tapioles, Sara Pardej, Elizabeth I. Pierpont, Julia Plank, Nancy Ratner, Katherine A. Rauen, J. Elliott Robinson, Les Rogers, Sarah E. Sheppard, Keir Shiels, David Stevenson, Dagmar Tiemens, Matthew Traylor, K. Nicole Weaver, Marielle Yohe, Tamar Green   +41 more
wiley   +1 more source

Resolution of Refractory Multifocal Atrial Tachycardia in Costello Syndrome Using Trametinib: A Case Supporting MEK Inhibitors as Targeted, Specific Antiarrhythmic

American Journal of Medical Genetics Part A, EarlyView.
ABSTRACT Arrhythmias affect approximately half of patients with Costello syndrome (CS, OMIM # 218040), with non‐reentrant atrial tachycardia being the most common. This case describes an infant with Costello syndrome carrying the pathogenic HRAS c.34G>A (p.G12S) variant who developed early‐onset, drug‐refractory multifocal atrial tachycardia (MAT ...
Vanina Taliercio, Annabelle Wilcox, Stacey Cole, Josue Flores Daboub, José E. Morales Moreno, Kasey G. Andrews, S. Yukiko Asaki, Thomas A. Pilcher, Martin Tristani‐Firouzi, Mary C. Niu, David Viskochil, Benjamin Hammond   +11 more
wiley   +1 more source

Short-term safety results from compassionate use of risdiplam in patients with spinal muscular atrophy in Germany

Orphanet Journal of Rare Diseases, 2022
Background The oral, selective SMN2-splicing modifier risdiplam obtained European approval in March 2021 for the treatment of patients ≥ 2 months old with a clinical diagnosis of 5q-associated spinal muscular atrophy (SMA) 1/2/3 or with 1–4 SMN2 gene ...
Andreas Hahn, René Günther, Albert Ludolph, Oliver Schwartz, Regina Trollmann, Patrick Weydt, Markus Weiler, Kathrin Neuland, Martin Sebastian Schwaderer, Tim Hagenacker, the Risdiplam Compassionate Use Program Group   +10 more
doaj   +1 more source

Superannuation Reimagined: Moving Beyond the Origins to an Indigenous Focus

Australian Journal of Social Issues, EarlyView.
ABSTRACT Retirement income systems, such as superannuation, are meant to be non‐discriminatory and consider disadvantage faced by members of society. There are significant differences between the life expectancies of Indigenous and non‐Indigenous peoples. The gap in life expectancies is not considered when determining when Indigenous peoples can retire.
Levon Ellen Blue, Peter Anderson, Mark Brimble   +2 more
wiley   +1 more source

The economic impact of 14 compassionate use programs for medicines in Italy, in the perspective of the National Health Service

Global & Regional Health Technology Assessment
Background: Compassionate use programs (CUP) for medicines allow patients, not recruited in trials, accessing medicines before marketing approval. The evidence on the economic impact of CUP is limited to one paper that did not include the possible long ...
Claudio Jommi, Federico Pantellini, Giovanni Giuliani, Marianna Cavazza   +3 more
doaj   +1 more source

The Cost of the National Disability Insurance Scheme: Australia's Print‐Media Discourse

Australian Journal of Social Issues, EarlyView.
ABSTRACT This paper examines the way that Australian newspapers have framed the cost of the National Disability Insurance Scheme (NDIS). Introduced in 2013, the NDIS represented a major change in Australia's disability support policy, moving for the first time to a nationwide universal insurance model.
Meera Chinnappa, Jennifer Smith‐Merry, Kuo‐yi Jade Chang   +2 more
wiley   +1 more source

Realising Aboriginal Community Controlled Approaches to Child Reunification

Australian Journal of Social Issues, EarlyView.
ABSTRACT Reunification rates for Aboriginal and Torres Strait Islander children in out‐of‐home care (OOHC) in Australia are critically low, even though reunification is the preferred permanency outcome for children following removal, and despite a range of mechanisms and strategies ostensibly to support effective reunification. To better understand the
B. J. Newton, Paul Gray, Kathleen Falster, Ilan Katz, Kyllie Cripps   +4 more
wiley   +1 more source